Astellas and Avexis Collaborate on Gene Therapy for Spinal Muscular Atrophy

Astellas Pharma Inc. (TSE: 4503) has announced a strategic partnership with AveXis, Inc., a Novartis company, to advance the development and commercialization of a gene therapy for Spinal Muscular Atrophy (SMA). The collaboration, aimed at expanding treatment options for this rare and debilitating neuromuscular disease, underscores Astellas’ commitment to addressing unmet medical needs in the field of genetic disorders.

Under the terms of the agreement, Astellas will obtain exclusive rights to collaborate with AveXis on the continued development of AVXS-101, a gene therapy for SMA. The partnership will focus on both the clinical development and the potential commercialization of this promising therapy in key global markets.

Breakthrough Gene Therapy for SMA

SMA is a rare genetic disorder caused by mutations in the SMN1 gene, leading to the degeneration of motor neurons, severe muscle weakness, and loss of motor function. It is one of the leading genetic causes of infant mortality, with an early onset of symptoms often resulting in significant health complications and, in many cases, premature death. AVXS-101 is an investigational one-time gene therapy designed to address the underlying cause of SMA by delivering a functional copy of the SMN1 gene to motor neurons, potentially halting disease progression or improving motor function.

The therapy has already shown promising results in clinical trials, demonstrating significant improvements in motor function for patients with SMA, particularly for those with the most severe forms of the disease. If successfully commercialized, AVXS-101 could provide a transformative treatment option for SMA patients, offering hope for those affected by the condition and their families.

Astellas' Commitment to Gene Therapy

The collaboration with AveXis marks a significant milestone in Astellas’ growing portfolio of gene therapies. "Astellas is excited to partner with AveXis on this innovative gene therapy for SMA," said Yoshihiko Hatanaka, President and CEO of Astellas. "This collaboration reflects our ongoing commitment to advancing transformative therapies that address the root causes of diseases and bring meaningful improvements to patients’ lives."

Astellas brings substantial expertise in developing and commercializing cutting-edge treatments, particularly in the field of rare diseases. The company’s experience in gene therapies, combined with AveXis’ leadership in SMA treatment development, positions the partnership to accelerate the availability of new, potentially life-changing options for SMA patients.

Terms of the Partnership

While the full financial details of the collaboration were not disclosed, the agreement includes both upfront payments and performance-based milestones, along with royalty payments based on future sales. Astellas will work closely with AveXis to progress AVXS-101 through further clinical development, regulatory approvals, and potential market entry.

The collaboration is expected to facilitate the global expansion of gene therapies for SMA, with a focus on bringing the treatment to patients in regions such as the U.S., Europe, and Japan, where Astellas has a strong commercial presence.

About Spinal Muscular Atrophy (SMA)

SMA is a genetic disorder that affects approximately 1 in 6,000 to 10,000 live births worldwide. It leads to progressive muscle weakness and atrophy, primarily affecting infants and young children, with varying degrees of severity. Without early intervention, SMA can lead to respiratory failure and loss of motor function. However, with early diagnosis and treatment, patients can experience significant improvements in motor skills, mobility, and quality of life.

About Astellas Pharma

Astellas Pharma Inc. is a global pharmaceutical company dedicated to improving the health of people around the world. With a focus on research and development in areas such as oncology, immunology, neuroscience, and rare diseases, Astellas is committed to bringing innovative therapies to patients. The company works to meet the unmet medical needs of patients, aiming to improve their quality of life and achieve better health outcomes.

About AveXis (now Novartis Gene Therapies)

AveXis, a Novartis company, is a leader in gene therapy with a focus on developing transformative treatments for patients with serious neurological genetic disorders. The company is particularly known for its work in SMA, where its groundbreaking gene therapy has shown to provide significant improvements in clinical outcomes. AveXis remains at the forefront of innovation in gene therapies for rare diseases.